Nevanimibe HCl for the Treatment of Classic CAH

Millendo Therapeutics

Status and phase: Terminated; Phase 2

Conditions: Congenital Adrenal Hyperplasia

Treatments: Drug: Nevanimibe hydrochloride

Study type: Interventional

Funder types: Industry

Identifiers

NCT03669549

ATR-101-202

Details and patient eligibility

About

This is a multicenter, intra-subject dose-titration open-label study of nevanimibe hydrochloride (HCl) for the treatment of classic congenital adrenal hyperplasia (CAH). Following a Screening Period of approximately 2-14 weeks, eligible subjects will enter a Baseline Period of approximately 2-8 weeks and then a 16-week Treatment Period. It is anticipated that the overall duration of the study per subject will range from 24-42 weeks.

Enrollment: 15 patients

Sex: All

Ages: 18 to 80 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Documented historical diagnosis of classic CAH due to 21-hydroxylase deficiency and/or historical documentation of elevated 17-OHP
• Chronic glucocorticoid replacement therapy for at least 6 consecutive months prior to screening
• Stable glucocorticoid and mineralocorticoid regimen for at least 4 weeks prior to screening and throughout the treatment period of the study

Exclusion criteria

• Nonclassic CAH
• Other causes of adrenal insufficiency
• HIV, hepatitis B, or hepatitis C
• AST or ALT >2x ULN, bilirubin or serum creatinine >1.5x ULN

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

---

15 participants in 1 patient group

Nevanimibe hydrochloride

Experimental group

Description:

Ascending dose level of oral nevanimibe hydrochloride beginning with 500 mg BID up to 2000 mg BID

Treatment:

Drug: Nevanimibe hydrochloride